Epidermolysis Bullosa SimplexPhase 2EBULONCT06509984
A 20-Week Study Assessing the Efficacy of Apremilast in Patients with EB Simplex Generalized
A Phase 2 study testing Apremilast treatment in people with Epidermolysis Bullosa Simplex and Genodermatosis, run by Centre Hospitalier Universitaire de Nice at 4 sites in France.
Trial summary
The goal of this clinical trial is to Assessing the Efficacy and Safety of Apremilast in Patients > 6 years of age with EB simplex generalized . The main question it aims to answer are: describe efficacity of this treatment. Participants will take treatments and have to use bullets during the study period. Read more about this study
- Who is it for?
- Ages 6 to 99 years
- Conditions the study is for
- Epidermolysis Bullosa Simplex, Genodermatosis
- What is being tested?
- Apremilast treatment
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 4 sitesFrance
- Study dates
- 6 Aug 2024to 1 Apr 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female patients 6 years or older
- Laboratory confirmed diagnosis of EBS-sev due to KRT5 or 14 mutation (autosomal)
- Mean daily number of new blisters >3.
- Subject/caregiver agrees not to use any topical therapies other than the investigator approved
Who cannot join
- - EBS lesions requiring oral therapy to treat an infection
- Use of any diacerein containing product within 6 months prior to Visit 1
- Use of systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Visit 1
- Use of systemic steroidal therapy within 30 days prior to Visit 1
- Use of any systemic product that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study assessments within 30 days prior to Visit 1
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
France4 sites, 4 recruiting
- Recruiting
CHU de Nice
Nice, France, 06000
Christine CHIAVERINI, 0492034702, chiaverini.c@chu-nice.fr
Thomas Hubiche, 0492034702, hubiche.t@chu-nice.fr
- Not yet recruiting
APHP Necker-Enfants-Malades
Paris, France, 75015
Christine Bodemer
- Not yet recruiting
APHP Hôpital Saint-Louis
Paris, France, 75018
Emmanuelle Bourrat
- Not yet recruiting
chu de Toulouse
Toulouse, 31059
Juliette MAZEREEUW -HAUTIER
About this study
Official title: EBULO. a 20-Week Multicentre, Open Study Assessing the Efficacy and Safety of Apremilast in Patients > 6 Years of Age with EB Simplex Generalized
The goal of this clinical trial is to Assessing the Efficacy and Safety of Apremilast in Patients > 6 years of age with EB simplex generalized . The main question it aims to answer are: describe efficacity of this treatment.
Participants will take treatments and have to use bullets during the study period.
Read the detailed description
The patient will have 7 visits. After Confirmation of inclusion criteria and no exclusion criteria, the study will be explain to the patient after signing the inform consent and be included in the study Treatment Period (Period 1); challenge period This period will be the first treatment period that will occur from 8 weeks. During this period the patient will see the doctor at the hospital, he will perform the study procedures The second period : dechallenge (period 2): All patients stop their treatment for 4 weeks.
The second treatment period - rechallenge (period 3) the patient will take the treatment for a second 8 weeks period At week 20 - End of study
At each visit:
The investigator at each visit compliance to the treatment and adverse events, Measure vital signs and perform clinical examination.
The patient will assess the different questionnaires, for the study
- Conditions
- Epidermolysis Bullosa Simplex, Genodermatosis
- Being tested
- Apremilast treatment
- Keywords
- Apremilast, Children, Pediatric
- Medical subject headings
- Epidermolysis Bullosa Simplex, Skin Diseases, Genetic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Apremilast treatmentDrug
treatment of apremilast
| Study group | Role | What they receive |
|---|---|---|
| apremilast treatment | Experimental | treatment by apremilast in two phases of challenge Receives: Drug: Apremilast treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
To describe efficacy of apremilast
number of new blister conting every day
Measured: weeks 20
Secondary outcomes (1)
Safety of apremilast treatment
number of SAE
Measured: weeks 20
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2024
- Study start6 Aug 2024
- Primary completion1 Oct 2025 (estimated)
- Study completion1 Apr 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Epidermolysis Bullosa SimplexStudies of the same condition, open and under way.
- More trials run by Centre Hospitalier Universitaire de NiceThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Nice
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06509984
- Sponsor's protocol ID
- 23-PP-10
- Other IDs
- 2023-508794-83-00, 2023
- Acronym
- EBULO
- Status last verified
- Feb 2025
- First submitted
- 15 Jul 2024
- Last updated
- 24 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Fontas E, Hubiche T, Maruani A, Moschoi R, Hussain S, Chiaverini C. Multicentre phase II open study assessing the efficacy and safety of apremilast in children and adults with epidermolysis bullosa simplex generalised: study protocol for the EBULO study. BMJ Open. 2025 Nov 12;15(11):e103194. doi: 10.1136/bmjopen-2025-103194.
PubMed 41224305
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- EBULO. a 20-Week Multicentre, Open Study Assessing the Efficacy and Safety of Apremilast in Patients > 6 Years of Age with EB Simplex Generalized
- ClinicalTrials.gov ID
- NCT06509984
- Acronym / sponsor ID
- EBULO / 23-PP-10
- Conditions
- Epidermolysis Bullosa Simplex, Genodermatosis
- Keywords
- Apremilast, Children, Pediatric
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire de NiceUniversity, hospital or other
- Study start
- 6 Aug 2024 (actual)
- Primary completion
- 1 Oct 2025 (estimated)
- Study completion
- 1 Apr 2026 (estimated)
- First posted
- 19 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to Assessing the Efficacy and Safety of Apremilast in Patients > 6 years of age with EB simplex generalized . The main question it aims to answer are: describe efficacity of this treatment.
Participants will take treatments and have to use bullets during the study period.
Detailed description
The patient will have 7 visits. After Confirmation of inclusion criteria and no exclusion criteria, the study will be explain to the patient after signing the inform consent and be included in the study Treatment Period (Period 1); challenge period This period will be the first treatment period that will occur from 8 weeks. During this period the patient will see the doctor at the hospital, he will perform the study procedures The second period : dechallenge (period 2): All patients stop their treatment for 4 weeks.
The second treatment period - rechallenge (period 3) the patient will take the treatment for a second 8 weeks period At week 20 - End of study
At each visit:
The investigator at each visit compliance to the treatment and adverse events, Measure vital signs and perform clinical examination.
The patient will assess the different questionnaires, for the study
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| apremilast treatment | Experimental | Drug: Apremilast treatmenttreatment by apremilast in two phases of challenge |
Who may qualify
- Ages
- 6 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Male or female patients 6 years or older
- Laboratory confirmed diagnosis of EBS-sev due to KRT5 or 14 mutation (autosomal)
- Mean daily number of new blisters >3.
- Subject/caregiver agrees not to use any topical therapies other than the investigator approved
Key exclusion criteria (5 listed)
- - EBS lesions requiring oral therapy to treat an infection
- Use of any diacerein containing product within 6 months prior to Visit 1
- Use of systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Visit 1
- Use of systemic steroidal therapy within 30 days prior to Visit 1
- Use of any systemic product that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study assessments within 30 days prior to Visit 1
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Apremilast treatmenttreatment of apremilast | Drug | apremilast treatment |
Primary outcome
To describe efficacy of apremilast
Time frame: weeks 20
number of new blister conting every day
1 secondary outcome
Safety of apremilast treatment
Time frame: weeks 20
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2025.
- Derived from this study: PubMed 41224305Fontas E, Hubiche T, Maruani A, Moschoi R, Hussain S, Chiaverini C. Multicentre phase II open study assessing the efficacy and safety of apremilast in children and adults with epidermolysis bullosa simplex generalised: study protocol for the EBULO study. BMJ Open. 2025 Nov 12;15(11):e103194. doi: 10.1136/bmjopen-2025-103194.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Apremilast treatment in people with Epidermolysis Bullosa Simplex and Genodermatosis, run by Centre Hospitalier Universitaire de Nice at 4 sites in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To describe efficacy of apremilast (time frame: weeks 20). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06509984, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Feb 2025.